Spotlight shines on injected RNA drug against hepatitis C amid pills-only frenzy

The New England Journal of Medicine published data from a promising mid-stage study of Santaris Pharma's microRNA therapy against hepatitis C, bringing media attention to this unique method of combating the liver-damaging virus as most of the focus in the field is on new oral antiviral drugs.

Prior to the prestigious NEJM publication on Wednesday, Horsholm, Denmark-based Santartis presented top-line data from the mid-stage trial of its hep C candidate, miravirsen, at The Liver Meeting in November 2011. The 36-patient study showed that HCV patients on the microRNA-targeting therapy experienced only mild side effects and four out of 9 patients on the highest dose of miravirsen monotherapy had undetectable levels of RNA associated with the virus after 5 weekly doses.

Miravirsen, which sequesters a microRNA linked with HCV replication, was the first candidate from the class of closely watched therapies to enter clinical trials. Santaris recently began a small Phase II study of miravirsen, aiming to recruit 10 patients who failed to respond to the standard treatment of interferon and ribavirin, Dr. Michael Hodges, Santaris' chief medical officer, told FierceBiotech in an interview. He also hopes to a start a 20-patient study in this tough-to-treat population to test the use of miravirsen in combination with ribavirin and Vertex Pharmaceuticals' ($VRTX) Incivek.   

Santaris wants to show that its hep C candidate has promise in difficult patient populations as the company seeks partners for developing miravirsen. Part of the challenge for the company is standing out among a crowded field of hep C players, including large companies such as Gilead Sciences ($GILD), AbbVie and Bristol-Myers Squibb ($BMY) focused on late-stage development of all-oral combo treatments.

"We continue to have dialogues with all the usual suspects in pharma who are interested in hepatitis C to see if they will partner with this program," Hodges told FierceBiotech.

Miravirsen could stand out from the pack with more data on its ability to effectively combat all 6 genotypes of hep C rather than just one or two, and Hodges noted that the mechanism of the therapy could overcome viral resistance to existing treatments.

- here's the release
- see the article from Science News

Special Report: Hep C pill race report 2012: Gilead, others rush toward pharma gold